A Phase II Pharmacodynamic Investigation of the Efficacy of Vorinostat to Induce Fetal Hemoglobin in Adults With Severe Sickle Cell Disease Who Have Not Benefitted From Prior Therapy
- Since we are looking for the highest dose of vorinostat that can be administered safely
without severe or unmanageable side effects in participants who have SCD, the
participants dose may change while they are enrolled in the study.
- Participants will receive an initial dose of vorinostat for the first cycle (or 4
weeks). If they tolerate this dose, they will receive a higher dose for the second
cycle. The dose will be increased a 2nd time if they tolerate the dose of the second
cycle. Participants will continue on that highest dose for as long as they can
tolerate it, or, for a maximum of 4 cycles. The maximum dose participants could
receive is 400mg a day, 3 times a week.
- Participants will be given a study drug diary for each study cycle to record each time
they take vorinostat and indicate if they have any changes in their health or in
medications they are taking.
- During the study participants will come to the clinic for visits with the study team
and may have some of the following tests: physical examination, EKG, blood tests, urine
tests and echocardiogram.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
To determine the efficacy of vorinostat when administered orally in inducing a 4% absolute increase or a 100% increase in fetal hemoglobin levels in subjects with severe sickle cell disease who have failed prior therapy.
Maureen Okam, MD, MPH
Brigham and Women's Hospital
United States: Food and Drug Administration
|Dana-Farber Cancer Institute||Boston, Massachusetts 02115|
|Brigham and Women's Hospital||Boston, Massachusetts 02115|
|Children's Hospital Boston||Boston, Massachusetts 02115|